L. Christopher-Stine, MD, MPH, Division of Rheumatology, Johns Hopkins University, Baltimore, Maryland, USA;
A. Ciesluk, MPH, G. Vandenberg, MPH, Modus Outcomes, Division of THREAD, Cambridge, Massachusetts, USA.
J Rheumatol. 2024 Dec 1;51(12):1198-1207. doi: 10.3899/jrheum.2023-1137.
Dermatomyositis (DM) symptoms negatively affect the quality of life of individuals living with the disease. Disease-specific, patient-reported outcome (PRO) instruments are needed to assess symptoms important to individuals with DM. This study aimed to conceptualize patient DM experience and disease activity definition to refine the development of the Dermatomyositis Disease Symptom Questionnaire (DM-DSQ), a novel PRO instrument capturing patient-reported symptoms.
An observational, qualitative study was conducted with 30 individuals with DM (aged ≥ 18 yrs) in the US. A 1-hour semistructured interview, including concept elicitation and cognitive debriefing, was conducted with each participant. Inductive coding was used to identify concepts; a saturation analysis was conducted to confirm sample size. Concepts from transcripts were used to refine the preliminary conceptual model and DM-DSQ items.
Concept elicitation analysis findings included disease symptoms (eg, muscle weakness) and functional impacts (eg, walking). The analysis achieved conceptual saturation; the first 5 interviews uncovered most of the concepts. During cognitive debriefing of the DM-DSQ, participants found the items relevant, comprehensive, and easily understood (except for "skin sensitivity in sunlight"). The revised DM-DSQ content appears preliminarily valid in the patient population surveyed, pending further additions and debriefing based on refinement of the preliminary conceptual disease model and items.
The DM-DSQ is being used in a phase II clinical trial and could become a valuable tool for studies evaluating PROs in patients with DM. Preliminary results indicate its content validity; extensive psychometric analysis using clinical trial data will determine its ability to capture symptoms for patients with DM.
皮肌炎（DM）症状会对患者的生活质量产生负面影响。需要使用特定于疾病的、患者报告的结局（PRO）工具来评估对 DM 患者重要的症状。本研究旨在构建患者 DM 体验和疾病活动的定义，以完善皮肌炎疾病症状问卷（DM-DSQ）的开发，这是一种新的用于评估患者报告症状的 PRO 工具。
在美国进行了一项观察性、定性研究，共纳入 30 名年龄≥18 岁的 DM 患者。每位参与者进行了 1 小时的半结构化访谈，包括概念发掘和认知审查。采用归纳编码识别概念；进行了一项饱和度分析以确认样本量。从转录本中提取概念用于完善初步概念模型和 DM-DSQ 项目。
概念发掘分析结果包括疾病症状（如肌肉无力）和功能影响（如行走）。分析达到了概念饱和度；前 5 次访谈揭示了大部分概念。在 DM-DSQ 的认知审查中，参与者认为项目相关、全面且易于理解（除了“阳光敏感的皮肤”）。修订后的 DM-DSQ 内容在调查的患者群体中初步具有有效性，但需要进一步添加内容并根据初步概念疾病模型和项目的完善进行审查。
DM-DSQ 正在一项 II 期临床试验中使用，可能成为评估 DM 患者 PRO 的有价值工具。初步结果表明其具有内容有效性；使用临床试验数据进行广泛的心理测量学分析将确定其用于捕捉 DM 患者症状的能力。
